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Detection of circulating miRNAs: Comparative analysis of extracellular vesicle-incorporated miRNAs and cell-free miRNAs in whole plasma of prostate cancer patients

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Circulating cell-free miRNAs have emerged as promising minimally-invasive biomarkers for early detection, prognosis and monitoring of cancer. They can exist in the bloodstream incorporated into extracellular vesicles (EVs) and ribonucleoprotein complexes.

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R E S E A R C H A R T I C L E Open Access

Detection of circulating miRNAs:

comparative analysis of extracellular

vesicle-incorporated miRNAs and cell-free

miRNAs in whole plasma of prostate cancer

patients

Edgars Endzeli ņš1 †, Andreas Berger1†, Vita Melne1,2, Cristina Bajo-Santos1, Krist īne Soboļevska1

, Art ūrs Ābols1

, Marta Rodriguez3, Daiga Šantare4

, Anastasija Rud ņickiha1

, Vilnis Lietuvietis1,2, Alicia Llorente3and Aija Lin ē1*

Abstract

Background: Circulating cell-free miRNAs have emerged as promising minimally-invasive biomarkers for early detection, prognosis and monitoring of cancer They can exist in the bloodstream incorporated into extracellular vesicles (EVs) and ribonucleoprotein complexes However, it is still debated if EVs contain biologically meaningful amounts of miRNAs and may provide a better source of miRNA biomarkers than whole plasma The aim of this study was to systematically compare the diagnostic potential of prostate cancer-associated miRNAs in whole plasma and in plasma EVs

Methods: RNA was isolated from whole plasma and plasma EV samples from a well characterised cohort of 50 patient with prostate cancer (PC) and 22 patients with benign prostatic hyperplasia (BPH) Nine miRNAs known to have a diagnostic potential for PC in cell-free blood were quantified by RT-qPCR and the relative quantities were

Results: Only a small fraction of the total cell-free miRNA was recovered from the plasma EVs, however the

EV-incorporated and whole plasma cell-free miRNA profiles were clearly different Four of the miRNAs analysed showed a diagnostic potential in our patient cohort MiR-375 could differentiate between PC and BPH patients when analysed in the whole plasma, while miR-200c-3p and miR-21-5p performed better when analysed in plasma EVs EV-incorporated but not whole plasma Let-7a-5p level could distinguish PC patients with Gleason score≥ 8 vs ≤6

Conclusions: This study demonstrates that for some miRNA biomarkers EVs provide a more consistent source of RNA than whole plasma, while other miRNAs show better diagnostic performance when tested in the whole plasma

Keywords: Prostate cancer, Cell-free miRNAs, Extracellular vesicles, Exosomes, Microvesicles, Biomarkers, Liquid biopsy

* Correspondence: aija@biomed.lu.lv

†Equal contributors

1 Latvian Biomedical Research and Study Centre, Ratsupites Str 1, k-1, Riga

LV-1067, Latvia

Full list of author information is available at the end of the article

© The Author(s) 2017 Open Access This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made The Creative Commons Public Domain Dedication waiver

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Circulating cell-free micro-RNAs (miRNAs) have emerged

as promising biomarkers for the development of

blood-based assays for early detection, prognosis and monitoring

of cancer In 2008, Mitchell et al demonstrated for the first

time that miRNAs are released from prostate cancer (PC)

cells into the bloodstream, where they exist in a remarkably

stable form [1] miRNAs were shown to remain stable after

incubation of plasma or serum at room temperature for up

to 24 h and to resist RNase A digestion, HCl and NaOH

treatment or multiple freeze-thaw cycles [1, 2]

Subse-quently, the levels of circulating miRNAs have been studied

in patients with various cancers, including PC, resulting in

the discovery of individual miRNAs or miRNA signatures

with diagnostic and/or prognostic value [3]

PC is the most frequently diagnosed cancer in males in

Europe and the United States [4, 5] Currently, the serum

PSA test is the most commonly used tool for organised

screening programs, opportunistic screening and

monitor-ing of PC [6] However, PSA is not cancer specific and the

high false-positive rate and low specificity leads to large

numbers of unnecessary prostate biopsies and emotional

morbidity [7] Furthermore, PC is characterised by a highly

heterogeneous course - one part of the patients develops a

high-grade disease with extracapsular spread and distant

metastases requiring aggressive treatment, while others

have a relatively indolent, slowly progressing disease that

could have been managed by active surveillance [8] The

current standard of care analyses, however, do not predict

whether a histologically proven tumour will give rise to a

clinically significant disease, leading to overtreatment of

indolent PC Hence, the greatest unmet clinical needs in

the management of PC are sensitive and reliable

non-invasive tools for differentiating between PC and benign

prostatic diseases, and between potentially fast progressing

PC requiring aggressive treatment and a relatively indolent

disease that can be managed by active surveillance

More than 20 studies investigating levels of cell-free

miRNAs in plasma or serum of PC patients have been

published up to date [9, 10] The majority of these studies

were focused on the identification of circulating miRNAs

that differentiate between patients with PC and benign

prostatic hyperplasia (BPH) or healthy controls Some of

these studies have shown remarkably high diagnostic

value For example, Chen et al identified a 5 miRNA panel

that could differentiate PC from BPH with an AUC of

0.924 and PC from healthy controls with an AUC of 0.860

[11] Some other studies have reported cell-free miRNAs

that differentiate between localised and metastatic

castra-tion resistant prostate cancer (mCRPC) or between

low-grade and high-low-grade PC For example, Mihelich et al

developed a“miRScore” that based on the serum levels of

14 miRNAs could predict absence of high-grade PC

among men with PC and BPH with a negative predictive

value of 0.939 [12] However, relatively few miRNA biomarkers have been validated by several independent studies, while many other miRNAs either have been reported in a single study or show conflicting results [3, 10] Therefore, the analysis of cell-free miRNAs is regarded

as a poorly reproducible technique [3, 13, 14]

Cell-free miRNAs circulating in the bloodstream have been found to be enclosed into extracellular vesicles (EVs) [15, 16], or to exist in a vesicle-free form associated with high-density lipoproteins [17], Ago2 protein [18, 19] or other RNA binding proteins [20] The majority of the studies has used whole plasma or serum as a source of cell-free miRNAs However, it has recently been hypothe-sised that cancer-derived EVs may be enriched with miRNA signatures reminiscent of their cell of origin, con-tain rare yet highly specific RNA biomarkers and protect their RNA cargo from degradation in the bloodstream and, therefore, the analysis of EV-enclosed miRNAs may be su-perior to whole plasma/serum analysis [10, 21, 22] Never-theless, to the best of our knowledge, a direct comparison

of miRNA detection assays in whole plasma and plasma EVs has not been reported so far

In this study, we evaluated the performance of 9 miRNA biomarkers previously reported to have a diagnostic or prognostic significance in PC by quantifying them in the whole plasma and plasma EVs in a cohort of 50 PC and

22 BPH patients

Methods

Study population and sample collection

Patients with PC and BPH were recruited between September 2011 and December 2013 at Riga East University Hospital and subsequently were followed up until December 2016 The diagnosis was established using standard of care diagnostic examinations and Gleason score was determined according to standard histopathological criteria by an experienced pathologist Pre-treatment blood samples were collected into EDTA-coated tubes and processed at room temperature within

2 h of blood draw Plasma samples were centrifuged twice for 10 min at 2000 g, aliquoted and stored at−80 °C until analysis The samples were deposited into the Latvian Genome Database Biobanking procedures were approved

by the Committee of Medical Ethics of Latvia and the use

of clinical samples for the research was approved by the Committee of Biomedical Ethics of Riga East University Hospital The blood samples were collected after the patients’ informed written consent was obtained

The following groups of patients were selected from the Database: PC with Gleason score ≥ 8 (Gleason high,

n = 24), PC with Gleason score ≤6 (Gleason low, n = 26) and BPH (absence of PC confirmed by histological examination of ultrasound-guided needle biopsies and

no change in the diagnosis within the follow-up period,

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n = 22) Clinical data of the study population are

provided in Table 1 In addition, plasma samples from 5

PC patients and 5 healthy controls were used for the

quality control of EV isolation

Isolation of extracellular vesicles

EVs were isolated from 400 μl of plasma using size

exclusion chromatography (SEC) SEC columns were

pre-pared by filling TELOS SPE columns (Kinesis, USA) with

10 ml (bed volume) of CL6B sepharose (GE Healthcare,

USA) Plasma samples were loaded on the columns and

gravity-eluted with PBS The eluate was collected in 12

sequential 0.5 ml fractions Each fraction was measured by

Zetasizer Nano ZS (Malvern, UK) and fractions

contain-ing particles larger than 30 nm were combined and

con-centrated to 100μl using Amicon Ultra 3 kDa centrifugal

filters (Merck, Millipore, Germany)

Transmission electron microscopy

Ten μl of EV suspension in PBS were applied to

300-mesh carbon coated copper EM grid and incubated for

5 min Then the samples were negatively stained with 1% uranyl formate (w/v) for 1 min, dried and examined using JEM-1230 transmission electron microscope (JEOL, USA)

Nanoparticle tracking analysis

Size distribution profile and concentration of EVs was de-termined using NanoSight NS500 instrument (Malvern, UK) EV samples were diluted 1000–25,000 fold in PBS to achieve particle concentration in range from 1×108 to 1×109particles/ml For each sample, five 30 s videos were recorded with the following settings: 25C, 0.944–0.948 cP,

1259 slider shutter, 366 slider gain, and 11 camera level The data analysis was performed with NanoSight NTA Software v3.1 Build 3.1.54 in the auto mode

Western blot

EVs and PC-3 cells (used as a positive control) were lysed in RIPA buffer (150 ml NaCl, 1% Triton X-100, 0.5% Na deoxycholate, 0.1% SDS, 50 ml Tris) and the protein concentration was assessed using Pierce™ BCA

Table 1 Clinical characteristics of the study population

Age (years)

Serum PSA (ng/ml)

Gleason score

Metastasis status

Cancer grade

Prostatitis

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Protein Assay Kit (Thermo Fisher Scientific, USA)

fol-lowing manufacturer’s instructions Thirty micrograms

of EV and cell proteins were mixed with Laemmli buffer

under reduction conditions, denatured for 5 min at 100 °C

and loaded on 10% SDS-PAGE gel Proteins were

electro-blotted to nitrocellulose membranes and the membranes

were blocked with 10% (w/v) fat-free milk and then

incu-bated with the following primary antibodies: anti-TSG101

(Abcam, # ab125011), Calnexin (Abcam, # ab22595), CD9

(Santa Cruz Biotechnology, # sc-13118) and β-actin

(Abcam, # ab8224) in 1:1000 dilution The blots were

washed and incubated with horseradish

peroxidase-conjugated goat anti-rabbit IgG F(ab’)2-HRP (1:2000)

(Santa Cruz, #sc-3837) or chicken anti-mouse IgG-HRP

(1:2000) (Santa Cruz, #sc-2962) secondary antibodies,

re-spectively Protein expression was visualized using

Western Blotting Detection Reagent kit (GE HealthCare

Lifesciences, Germany)

Enzymatic treatment

Prior to RNA extraction, EVs samples were treated with

1 mg/ml proteinase K (Thermo Fisher Scientific, USA) for

30 min at 37 °C Proteinase K was inactivated by

incubat-ing the samples for 10 min at 65 °C Then the samples

were treated with 10 ng/μl RNase A (Thermo Fisher

Scientific, USA) for 15 min at 37 °C

RNA extraction

RNA was extracted from EV and whole plasma samples

using miRNeasy Micro Kit (Qiagen, USA) according to

the manufacturer’s instructions with slight modifications

of the protocol Briefly, 5 volumes of QIAzol Lysis

Reagent were added to each sample Subsequently,

sam-ples were spiked with 1μl of UniSp6 (Exiqon, Denmark),

which was used as a normaliser in downstream analysis

After adding 1 volume of chloroform samples were

cen-trifuged for 15 min at 12000 g at 4 °C and the aqueous

phase was transferred to a new tube Then, 1.5 volumes

of 100% ethanol were added to each sample and the

mixture was loaded onto a MinElute spin column

Columns were centrifuged at 1000 g for 30 s at room

temperature in each round until entire sample was

loaded RNA was eluted in 15 μl of RNase-free water

using low-bind tubes The quantity and quality of RNA

was assessed using Agilent 2100 Bioanalyzer and RNA

6000 Pico Kit (Agilent technologies, # 5067–1513)

RT-qPCR analysis

One third of each RNA sample isolated from EVs and

whole plasma was reverse-transcribed using miRCURY

LNA Universal cDNA Synthesis kit II (Exiqon)

accord-ing to the manufacturer’s protocol cDNA reaction

mix-tures were diluted 1:40 and 4 μl were used for qPCR

reactions qPCR was carried out using microRNA LNA

PCR primer sets and ExiLENT SYBR Green master mix (Exiqon) according to the manufacturer’s protocol on ViiA

7 Real-Time PCR system (Thermo Fisher Scientific)

Statistical analysis

Ct values were averaged between duplicates and normalized against UniSp6 spike-ins by subtracting them from average spike-in Ct values in the same samples, resulting in log2 relative quantities (log2 RQ’s) The statistical analyses were performed with GraphPadPrism 5 (GraphPad, USA) A non-parametric Mann-Whitney U test was used to com-pare the RQ values of each miRNA between the groups of samples Multiple testing correction was done by false discovery rate (FDR) estimation and adjusted (adj.) P-value

of ≤0.05 was considered to be significant To assess the diagnostic potential, the area under the ROC curve (AUC) was calculated for each miRNA

Results

Selection of miRNA biomarkers

Nine miRNAs, whose levels in plasma or serum have been reported to have a diagnostic or prognostic significance in

PC in at least two independent studies, were selected for this study Studies showing their relevance for the diagnosis

or prognosis of PC are summarised in Table 2 MiR-21-5p, miR-200c-3p, miR-210-3p and miR-375 have been shown

to be increased in the blood of PC patients as compared to BPH or healthy controls consistently by two or more stud-ies, while miR-30c-5p and miR-223-3p were found to be consistently decreased in the blood of PC patients Incon-sistent findings have been reported for Let-7a-5p, miR-141-3p and miR-106a-5p

Yield and purity of EVs

In order to compare the levels of the selected miRNAs in plasma EVs and whole plasma, each plasma sample was divided into two 400 μl aliquots – one was used for the isolation of EV-incorporated RNA, while another was used directly for the isolation of cell-free RNA from whole plasma according to the workflow shown in Fig 1a

To assess the yield and purity of EVs, EV samples from

5 PC patients and 5 healthy controls (not included in the miRNA analysis) were characterised by transmission electron microscopy (TEM), nanoparticle tracking analysis (NTA) and Western blot analysis TEM images revealed that the majority of particles were ranging in size from 25 to 60 nm that corresponds to the size of exosomes (Fig 1b) However, as it has been shown that SEC-based EV isolation methods do not result in lipoprotein-free EV preparations [23], it cannot be excluded that a fraction of the particles are lipoproteins NTA showed that the concentrations of EVs range from 3.14×1010 to 1.27×1012 particles per ml of plasma (Fig 1c) The EV count was slightly increased in plasma

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Table 2 Circulating cell-free miRNA biomarkers for prostate cancer

miRNA Expression in PC tissues Level in blood

type

Patient groups and sample size Direction Normalisation Ref

Let-7a-5p

Down in PC vs adj.

Normal tissues

[ 45 ] Serum PC (n = 75), BPH (n = 27) Down in PC RNA input and

miR-16, miR-425

[ 52 ] Down in PC vs BPH [ 44 ] Serum High grade PC (n = 50),

low grade PC (n = 50), BPH (n = 50)

Down in high grade PC

vs low grade PC, BPH

RNA input and spike-ins

[ 12 ]

Serum Disseminated PC (n = 20),

BPH (n = 13)

Up in disseminated PC Spike-in and

miR-320a

[ 37 ]

miR-21-5p

Up in PC vs adj Normal

(n = 10)

[ 55 ] Plasma mCRPC (n = 25, pooled),

LPC (n = 25, pooled)

Similar in PC and adj.

Normal tissues (n = 36)

[ 56 ] Serum ADPC (n = 20), HRPC

(n = 10), LPC (n = 20), BPH (n = 6)

Up in HRPC vs ADPC, LPC U6 snRNA [ 42 ]

Up in PC vs

normal tissues

[ 57 ] Plasma PC (n = 51),

HC (n = 20)

miR-30c-5p

Up in PC vs adj Normal

epithelium (n = 37)

[ 58 ] Serum High grade PC (n = 50),

low grade PC (n = 50), BPH (n = 50)

Down in high grade PC

vs low grade, BPH

RNA input and spike-ins

[ 12 ]

Up in PC vs

normal tissues

[ 57 ] Plasma PC (n = 80), BPH (n = 44),

HC (n = 54)

Down in PC vs BPH, HC U6 snRNA [ 11 ] Serum PC (n = 36), HC (n = 12) Down in PC RNA input [ 51 ]

miR-106a-5p

Up in PC vs

normal tissues

[ 57 ] Serum High grade PC (n = 50),

low grade PC (n = 50), BPH (n = 50)

Down in high grade PC RNA input and

spike-ins

[ 12 ]

Serum PC (n = 36), HC (n = 12) Up in PC RNA input [ 51 ]

miR-141-3p

Up in mPC, PC vs

normal tissues

[ 53 ] Serum High grade PC (n = 50),

low grade PC (n = 50), BPH (n = 50)

Detectable in <50%

of patients

RNA input and spike-ins

[ 12 ]

Up in PC vs BPH [ 52 ] Serum PC (n = 75), BPH (n = 27) Up in PC RNA input and

miR-16, miR-425

[ 52 ]

Up in BCR after RP

vs no BCR after RP

[ 59 ] Plasma mCRPC (n = 25, pooled),

LPC (n = 25, pooled)

Serum mCRPC (n = 26), low-risk LPC

(n = 28)

Up in PC (n = 36)

vs normal tissue

(n = 36)

[ 54 ] Plasma EVs PC (n = 78), HC (n = 28) Up in PC Spike-ins [ 38 ] Serum

EVs

mPC (n = 47), non-recurrent

PC (n = 72)

Up in mPC

Serum 71 PC: N1 (n = 48), N0 (n = 23),

GS ≥8 (n = 29), GS = 7 (n = 42) Up in N1 PC vs N0 PC;Up in GS ≥ 8 vs GS = 7 Spike-ins [54] Plasma mPC (n = 25), LPC (n = 26) Up in mPC vs LPC; Similar

in PC and HC

RNU1A snRNA [ 43 ] Serum mPC (n = 25), HC (n = 25) Up in mPC Spike-ins [ 60 ] Serum PC (n = 54), non-malignant

(n = 79)

Up in higher GS; Similar in

PC and non-malignant

RNU1 –4 and SNORD43

[ 61 ]

miR-200c-3p

Up in PC vs

normal tissue

[ 62 ] Plasma mCRPC (n = 25, pooled),

LPC (n = 25, pooled)

Serum mCRPC (n = 25), HC (n = 25) Up in mCRCP Spike-ins [ 41 ]

miR-210-3p

Up in PC vs BPH [ 44 ] Serum PC (n = 31), BPH (n = 13) Up in PC Spike-in and

miR-320a

[ 37 ] Serum mCRPC (n = 21), HC (n = 20) Up in mCRCP Spike-ins [ 41 ]

miR-223-3p

Up in PC vs adj.

Normal tissues (n = 10)

vs low grade, BPH

RNA input and spike-ins

[ 12 ]

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from PC patients as compared to the healthy controls

(mean count in PC 7.08×1011 vs 4.15×1011 in healthy

controls), although the difference didn’t reach statistical

significance in our sample set The size distribution

analysis showed that the diameter for the majority of

particles was in the range from 50 to 150 nm with a

minor fraction reaching ~230 nm (Fig 1d), which is

somewhat inconsistent with the TEM results This

discrepancy likely has arisen due to the difference in the

minimum detectable EV size between both techniques

[24] and /or shrinking of EVs during fixation for TEM

[25] Western blot analysis showed that the EVs were

positive for typical EV markers TSG101 and CD9, and

negative for the endoplasmic reticulum protein Calnexin

(Fig 1e) Taken together, these results show that the EV

isolation method used in this study results in a relatively

high yield of exosome-enriched EV preparations without

detectable contamination of intracellular components

RNA profiles in EVs and whole plasma

As it has been suggested that EVs may associate with

lipoproteins or protein complexes that carry cell-free

miRNAs and protect them from degradation [18, 26], we

first tested the effect of proteinase K and RNase A

treat-ment on the miRNA levels in plasma EVs from three

healthy individuals (Fig 2a) Treatment of EVs with

RNase A alone reduced the relative quantity (RQ) values

by 15.5 to 43.6% for different miRNAs, while the

treatment with proteinase K prior to RNase A resulted

in the reduction of RQs by 50.4 to 69.3% This suggests that the proteinase K treatment is required for efficient removal of extra-vesicular RNA Therefore, in order to study the intraluminal miRNAs, all EV preparations were treated with proteinase K and RNase A prior to the RNA extraction RNA was extracted from EVs and whole plasma using miRNeasy Micro kit, which is designed for isolation of total RNA from small amounts

of sample Typical RNA profiles obtained by Bioanalyzer from whole plasma and EVs are shown in Fig 2b The profiles show the presence of small RNA peaks of 25 to

200 nt both in whole plasma and EVs, while 18S and 28S rRNA peaks are present in whole plasma and EVs without the enzymatic treatment (not shown) but not in the treated EVs, thus suggesting that the majority of rRNA is bound to the surface of EVs

Relative abundance of EV-incorporated miRNAs

An equal proportion (one third) from the total RNA amount obtained from the EV and whole plasma samples of PC and BPH patients was used for the RT-qPCR analysis of the 9 selected miRNA biomarkers Spike-ins were used to control for a variation in RNA extraction, cDNA synthesis and PCR efficiency and they typically varied less than by 1 Ct In order to assess the relative abundance of EV-enclosed miRNAs, a ratio between EV-enclosed and total cell-free miRNAs in

Table 2 Circulating cell-free miRNA biomarkers for prostate cancer (Continued)

miRNA Expression in PC tissues Level in blood

type

Patient groups and sample size Direction Normalisation Ref High grade PC (n = 50),

low grade PC (n = 50), BPH (n = 50)

Up in PC vs

normal tissues

[ 57 ] Serum PC (n = 36), HC (n = 12) Down in PC RNA input [ 51 ] miR-375 Up in mPC, PC

vs normal tissues

[ 53 ] Plasma EVs

CRPC (n = 100) High miRNA level

associated with poor OS

RNA input and miR-30a-5p, miR-30e-5p

[ 39 ]

Serum PC (n = 31), BPH (n = 13) Up in PC Spike-in and

miR-320a

[ 37 ]

Up in PC (n = 36) vs

normal tissue (n = 36)

[ 54 ] Plasma mCRPC (n = 25, pooled),

LPC (n = 25, pooled)

Serum mCRPC (n = 26), low-risk

LPC (n = 28)

Serum EVs

mPC after RP (n = 47), non-recurrent PC after

RP (n = 72)

Serum 71 PC: N1 (n = 48), N0

(n = 23), GS ≥8 (n = 29),

GS = 7 (n = 42)

Up in N1 PC vs N0 PC;

similar in GS ≥ 8 and GS = 7 Spike-ins [54]

ADPC androgen-dependent prostate cancer, BCR biochemical recurrence, BPH benign prostatic hyperplasia, CRPC castration resistant prostate cancer, EVs extracel-lular vesicles, HC healthy control, HRPC hormone-refractory prostate cancer, LPC localized prostate cancer, mCRPC metastatic castration resistant prostate cancer, mPC metastatic prostate cancer, PC prostate cancer, RP radical prostatectomy

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whole plasma was calculated (Fig 3a) The results

showed that only a small fraction of the cell-free miRNA

was retrieved from the EVs However, the EV-enclosed

fraction was not uniformly low – it varied from 6.36%

for Let-7a-5p to 0.65% for miR-210-3p Spearman

correlation analysis revealed only weak to moderate

correlation between EV-enclosed and whole plasma

cell-free miRNAs (Table 3) As an example, a paired dot plot

in Fig 3b shows the discordance in the Let-7a-5p levels

in EVs and whole plasma from the same patients These data support the idea that EV-enclosed miRNA profile dif-fers from cell-free miRNA profile in the whole plasma Clearly, the size of the EV-enclosed miRNA fraction depends on the efficacy of the EV isolation method and the obtained ratios are not expected to represent the EV-enclosed: EV-free miRNA ratio However, the NTA

Fig 2 Effects of proteinase K and RNase A treatment on the relative quantity of EV-incorporated miRNAs and RNA profiles in whole plasma and EVs a RT-qPCR analysis of miRNA levels in EVs treated with RNase A alone or with a combination of proteinase K and RNase A relatively to untreated EVs Bars show the mean percentage in EVs from 3 healthy individuals b A representative RNA profile from whole plasma and EVs treated with proteinase K and RNase A obtained by Bioanlyzer RNA 6000 Pico chip

Fig 1 Workflow of the study and characterisation of plasma EVs a Workflow of the study b Representative transmission electron microscopy image of plasma EVs c Quantification of EVs isolated from plasma of PC patients and healthy controls (HC) by nanoparticle tracking analysis d Average size distribution of EVs isolated from plasma of PC patients and healthy controls e Western blot analysis of EV markers (TSG101, CD9), endoplasmic reticulum protein Calnexin and β-actin in plasma EVs isolated from two healthy individuals and PC-3 cells (as a positive control)

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data showed that the EV count recovered in this study

was similar or even higher than that reported by other

studies [27–30], therefore we assume that the EV yield

in our study is representative of that obtained by the

current standard EV isolation techniques

These results show that although only a small fraction of

the total cell-free miRNA present in plasma was recovered

from EVs, the EV-incorporated miRNA profile is clearly

dif-ferent from that in the whole plasma

Diagnostic potential of EV-enclosed and total cell-free

miRNAs

To assess the diagnostic potential of the selected miRNAs,

their relative quantity in EVs and whole plasma was

com-pared between patients with PC (n = 50) and BPH

(n = 22) Three of the 9 miRNAs tested showed a

diagnos-tic value in our sample set (Fig 4) MiR-375 was

signifi-cantly increased in PC patients as compared to BPH (FDR

adj p = 0.03) and had an AUC of 0.68 (95% CI: 0.54–0.83,

p = 0.01), when tested in the whole plasma The same

ten-dency was observed for EV-enclosed miR-375, however it

didn’t reach statistical significance On the contrary,

miR-200c-3p and miR-21-5p could differentiate between PC and BPH better when tested in EVs than in the whole plasma (AUC of 0.68, p = 0.01 and 0.67, p = 0.02, respect-ively, when tested in EVs and AUC of 0.62, p = 0.12 and AUC of 0.61, p = 0.16, respectively, when tested in whole plasma) The levels of the other miRNAs were not signifi-cantly different in PC samples compared to BPH neither

in EVs nor in whole plasma

Next, we investigated the association of EV-enclosed and whole plasma miRNA levels with PC aggressiveness

We found that the level of Let-7a-5p was significantly decreased in EVs from PC patients with high Gleason score (≥8) compared to low Gleason score (≤6) and it could differentiate between these groups with AUC of 0.68 (95% CI: 0.52–0.84, p = 0.03) (Fig 5) Although the same tendency was observed in whole plasma, the stand-ard deviation was larger and statistical significance was not reached No other miRNA could differentiate between PC patients with high and low Gleason scores Finally, none of the miRNAs was associated with the presence of histologically confirmed prostatitis in PC and BPH patients, thus showing that the alterations in the miRNA levels are not due to prostatic inflammation

Discussion

Cells can release miRNAs to the extracellular space either incorporated into EVs [31, 32] or in a vesicle-free form bound to various protein and lipoprotein com-plexes [17–20] Quantification of these miRNAs in blood from cancer patients may offer new opportunities for diagnosis, prognosis, monitoring of treatment response and early detection of recurrence in a minimally invasive way However, human blood contains a complex mixture

of miRNAs derived from various cell types and, there-fore, robust quantification of cancer-derived cell-free miRNAs has turned out to be a challenging task [14] Currently, it is still debated if the EV-based miRNA

Fig 3 Relative abundance of EV-incorporated miRNAs a Ratio between EV-incorporated and total cell-free miRNAs in whole plasma Bars represent the mean ratios in groups of patients with PC and BPH b A paired dot plot shows the ranking of PC patients according to Let-7a-5p levels in EVs and whole plasma; lines connect the samples from the same individual

Table 3 Spearman correlation coefficients of EV-enclosed and

whole plasma miRNAs

miRNA Spearman r 95% confidence interval p value

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detection assays are superior to the whole plasma-based

assays miRNA profiles in cancer-derived EVs have been

found to be reminiscent of their cell-of-origin [31, 33],

though due to selective RNA sorting mechanisms they

may be enriched or depleted of some specific miRNAs

[34] The EV membrane protects the RNA cargo from

degradation in the bloodstream and the intraluminal

RNA content is thought to be relatively stable, therefore

EVs may provide a more consistent source of miRNA

biomarkers than whole plasma [15, 30] On the other

hand, it has been calculated that there is far less than

one molecule of a given miRNA per EV [35], which

raises the question of whether all EVs contain miRNAs

and if the amounts are biologically meaningful

Moreover, it can be argued that the EV isolation step may introduce a higher variation and result in a low RNA yield that in turn would lead to lower sensitivity, higher standard deviations and poor reproducibility of the EV-based miRNA assays as compared to whole plasma assays

Here, we have performed a systematic comparison of miRNA levels in whole plasma and EVs isolated from the same plasma samples in a well-characterised cohort of PC and BPH patients Our results show that EV-incorporated miRNA constitutes only a minor fraction of whole plasma miRNA This is in line with a study by Chevillet et al showing that exosome fractions contained a small minor-ity of the miRNA content of plasma [35] Nevertheless,

Fig 4 Circulating miRNA levels in patients with BPH and PC Scatter plots show the log2RQ values of each miRNA tested in EVs and in whole plasma FDR-adjusted p values are show at the top of each graph Area under the ROC curve (AUC), 95% confidence interval and p value for differentiating between PC and BPH is shown below each graph

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the miRNA levels in EVs and whole plasma were poorly

correlated, and the EV-incorporated and whole plasma

miRNA profile was clearly different This finding is

con-sistent with a NGS-based study by Cheng et al that

com-pared small RNA profiles in EVs, plasma and serum of 3

healthy individuals and showed that the miRNA levels

dif-fer remarkably between plasma and serum EVs and

between EVs and cell-free plasma and serum [30]

Three out of 9 miRNAs analysed could differentiate

be-tween PC and BPH patients in our cohort MiR-375

showed a better diagnostic performance when tested in

whole plasma as compared to EVs MiR-375 is an

onco-genic miRNA that is overexpressed in tumours with high

Gleason score and more advanced pathological stage [36]

Increased plasma or serum levels of miR-375 in patients

with PC vs BPH or metastatic CRPC vs localised PC have

been reported before in several studies (Table 2), and the

AUC obtained in our study was similar to that reported

be-fore [37] MiR-375 had one of the lowest EV to whole

plasma ratios among the miRNAs analysed in this study

and it was undetectable in a significant portion of EV

sam-ples It still may have diagnostic properties in cases where it

is detectable, though proving its diagnostic value would

re-quire a larger cohort of samples Two studies have reported

the presence of miR-375 in blood EVs from PC patients

Bryant et al showed that its level is increased in serum EVs

from patients with metastatic PC as compared to

non-recurring PC [38], and Huang et al reported that high

EV-miR-375 level is associated with a poor prognosis in CRPC

[39] Hence, increased levels of EV-incorporated miR-375

appear to be associated with metastatic disease As only 3

of the patients in our cohort had a metastatic disease at the

time of the blood draw, we reasonably detected it only in a

minority of PC patients in our cohort Moreover, as these

studies did not describe treatment of EVs with proteinase

K, it is possible that the EV preparations also contained

protein-bound miRNAs co-isolated with EVs

On the contrary, EV-incorporated 200c-3p and miR-21-5p showed better diagnostic performance than in whole plasma Increased plasma or serum levels of miR-200c-3p have been found before in patients with metastatic CRPC

as compared to localised PC or healthy controls [40, 41] Similarly, miR-21-5p has been reported to be increased in plasma or serum of patients with PC as compared to healthy controls and patients with CRPC as compared to localised PC [40, 42, 43] However, to the best of our know-ledge, an association of EV-incorporated miR-200c-3p and miR-21-5p with PC has not been reported before Hence, our study shows for the first time that EVs provide a better source for testing these miRNAs as PC biomarkers than whole plasma

The only miRNA biomarker that could differentiate between PC patients with high vs low Gleason score was EV-incorporated Let-7a-5p, whose level was decreased

in patients with Gleason score≥ 8 This is in line with a study by Mihelich et al showing that serum levels of Let-7a were decreased in PC patients with Gleason 4 + 5 grade tumours as compared with Gleason grade 3 [12] Our study, though, shows that the whole plasma and EV levels of Let-7a-5p are poorly correlated and that EV-incorporated Let7a-5p level is more informative than Let7a-5p in whole plasma

The cellular origin of circulating miRNAs is unclear Although it seems likely that oncogenic miRNAs such as miR-375, miR-200c-3p and miR-21-5p that are overex-pressed in PC tissues are released in the bloodstream from the tumour tissues, direct evidence for this is still lacking On the contrary, Let-7a-5p is a tumour suppres-sive miRNA that is downregulated in PC tissues as com-pared to normal or BPH tissues [44, 45] Hence, the decrease in Let-7a-5p plasma level in patients with aggressive PC is unlikely to be due to the release from cancer tissue More plausibly, lower expression level or reduced release of Let-7a-5p is genetically associated

Fig 5 Circulating Let-7a-5p levels in PC patients with low and high Gleason score Scatter plots show the log2RQ values of Let-7a-5p tested in EVs and in whole plasma of patients with Gleason score ≥ 8 (PC GH) and Gleason score ≤6 (PC GL) The mean log2RQ values and standard deviation is shown above each scatter plot Area under the ROC curve (AUC), 95% confidence interval and p value for differentiating between PC patients with high and low Gleason score is shown below each graph

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